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T.P.1 05 Antisense oligonucleotide design for therapeutic antisense-mediated exon skipping for Duchenne muscular dystrophy

✍ Scribed by A. Aartsma-Rus; C.L. de Winter; W.E. Kaman; A.A.M. Janson; J.C.T. van Deutekom

Book ID: 116792597
Publisher: Elsevier Science
Year: 2006
Tongue: English
Weight: 43 KB
Volume: 16
Category: Article
ISSN: 0960-8966
DOI: 10.1016/j.nmd.2006.05.141

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## Abstract ## Background Antisense‐mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne